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A drug that could raise IQ? Balance Therapeutics raises $18M, conducting Down Syndrome trials

Awardee Story A drug that could raise IQ? Balance Therapeutics raises $18M, conducting Down Syndrome trials
Date: Mar 15, 2015

Source: MedCityNews ( click here to go to the source)

Stanford spinout Balance Therapeutics just raised $18 million for its therapy that could raise the IQ of people with intellectual disability. It’s using a small molecule drug that has been around for decades -- GABA-A antagonists -- but rationalizes the approach by delving deeper into the molecular underpinnings of what causes diseases like Down Syndrome and dementia.

And it could improve the IQ of a person with Down Syndrome 10 to 20 percent.

The financing comes from 22 investors, according to a regulatory filing. A company representative confirmed this is new funding, and will release details of the financing next week. It held a Series A round in 2011, with participants including Mohr Davidow Ventures, Pappas Ventures, Stanford University and individual investors. It’s also received significant R&D support from the NIH, Balance says on its website.
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Close examination of the molecular basis of intellectual disability has been quite revealing: Looks like the learning and memory deficits in conditions ranging from Down Syndrome to Alzheimer’s might be caused by a similar mechanism in the brain.

Neurotransmitters fire across synapses in the brain, allowing individuals to transmit and process information. A dampening of chemical and electrical signals may be the root cause for mental impairment, Stanford researchers have found.

Balance Therapeutics’ approach is to help restore the balance between inhibition and excitation of the synapses -- increasing brain plasticity. This, in turn, could help folks with intellectual impairment catch up on memory and learning tasks.

Last year, Bloomberg wrote about how such drugs -- including Balance’s -- could help children with Down Syndrome learn. When testing an old GABA-blocking drug called PTZ in mice, the researchers found that after 17 days the treatment, the Down mice had normalized -- for two whole months. The results were published in 2007 in Nature Neuroscience.

In a phone interview, Stanford University neurobiologist Craig Garner said of the animal results: “It was blood amazing. It was shocking how well it worked.”

Garner said he tried for years without success to get drug companies that had been pursuing GABA-blocking medicines for treating memory loss in the elderly to be interested in Down syndrome. In late 2009, he gave up and co-founded Balance Therapeutics with Lyndon Lien, a former Elan Corp. executive, and another scientist, Dan Wetmore.

Most recently, Balance Therapeutics has been conducting a 90-patient Phase 2 trial in Australia. Wagering the $18 mill is to kick off phase three.

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